China, Gene Editing

In a landmark first, Chinese scientist claims birth of genetically modified babies — and all hell breaks loose

A Chinese researcher is claiming that he created the first-ever genetically edited babies using CRISPR/Cas9 tools that have now become a common feature in labs around the world. And the news triggered a tempest in scientific circles the world over as researchers who have been using gene-editing tech to refine plants and forge new therapies try to puzzle out the stunning —if true — development featured on YouTube.

Several experts — including co-inventor Feng Zhang — criticized the experiment, raising potential safety issues that could arise as a result of the genetic tinkering. And Rice University, where a professor was reportedly involved, is investigating.

The scientist who claimed credit for the work, though, offered a sunny perspective on YouTube.

“Two beautiful Chinese girls named Lulu and Nana came crying into the world as healthy as any other babies a few weeks ago,” said Shenzhen-based researcher Jiankui He in the YouTube video and WeChat post. But this was no average birth. The researcher’s unverified claim is that the twins’ embryos have been genetically engineered with CRISPR to decommission CCR5, a gene used by HIV as a back door into a cell.

Ahead of an international conference on gene editing expected to commence on Tuesday in Hong Kong, He said he had altered embryos for seven couples during fertility treatments, with one pregnancy resulting thus far. But the legitimacy of the project is being investigated. The Southern University of Science and Technology in Shenzhen, with which He is affiliated, issued a statement saying it was “deeply shocked” and unaware of his research project which they consider a “serious violation of academic ethics and standards.” The university added He has been on leave without pay since February.

He has claimed his aim was not to cure or prevent an inherited disease, but to confer a trait that is naturally common in parts of Northern Europe — an ability to resist an HIV infection from the AIDS virus. But the choice to edit this CCR5 gene, the blockade of which may also be effective in thwarting cholera and smallpox, immediately triggered an online uproar over the use of CRISPR to alter DNA in a way that could be passed down for generations to come.

MIT Technology Review’s Antonio Regalado pointed out this could be particularly controversial because there are easier and cheaper ways to prevent HIV infection, or indeed suppress it. Editing embryos during IVF will also be expensive and involve technology out of reach for poorer pockets of the world where HIV is ubiquitous.

Some are also suggesting the story raises other thorny questions. Imperial College investigator Tom Ellis noted:

Although there is scientific consensus that gene editing should not be employed to make “designer babies” endowed with enhanced physical features or intellectual traits, the jury is out on to what degree science should interfere with nature to prevent, treat or cure disease.

In addition to China, laboratory research is underway in Sweden and the UK, investigating the potential of gene-editing in human embryos. But in the United States it’s a politically charged proposition that has won the narrow endorsement of the National Academy of Sciences, which last year recommended that germ-line modification of humans was justified in some circumstances, such as preventing the birth of children with serious diseases. This recommendation will likely fall on deaf ears, as sections of the public vehemently oppose such interference on religious grounds. In fact such modifications are practically out of the question — with laws in place prohibiting the FDA from even considering proposals to create genetically-edited offspring.

The promise of CRISPR/Cas9 editing has long been heralded. However, experimentation with the procedure has yielded significant safety concerns. Data presented earlier this year suggest that the tool, which is essentially a pair of molecular scissors, may inadvertently increase cancer risk in some cells, or introduce accidental mutations — issues that could hamper the development of gene-editing therapies championed by companies such as CRISPR Therapeutics $CRSP, Editas Medicine $EDIT and Intellia Therapeutics $NTLA.

“The genetic editing of a speck-size human embryo carries significant risks, including the risks of introducing unwanted mutations or yielding a baby whose body is composed of some edited and some unedited cells. Data on the Chinese trial site indicate that one of the fetuses is a ‘mosaic’ of cells that had been edited in different ways,” Regalado underscored in his article.

He’s project involved couples in which the men had HIV but the women did not, and the goal was to prevent their children from suffering the same fate.

According to the AP report, He said that in one twin, both copies of the intended gene had been altered, while in the other twin, just one copy had been disabled — and that there was no evidence of harm to any other genes. Humans with one copy of the gene can still be infected with HIV.

The editing occurred during IVF — first, the sperm was separated from the semen where HIV is known to linger. Then, a single sperm was placed into a solitary egg to create an embryo when the gene-editing tool was employed. Couples recruited to the study were given free fertility treatment in return for their participation and offered the choice to use either edited or non-edited embryos for pregnancy attempts. Overall, 16 of 22 embryos were edited, and 11 embryos were used in 6 implant attempts before the twin pregnancy was realized, He told the AP.

He, who is also a founder of a DNA sequencing company Direct Genomics, obtained informed consent from participants calling the study an “AIDS vaccine development project.” However, in his application form seeking ethical approval, he dubbed it a CCR5 gene editing project.

Press reports tied his work to Rice University’s Michael Deem, who now will have to answer for what, exactly, they did. Rice University was quick to launch its own probe. They noted:

Recent press reports describe a case of genomic editing of human embryos in China. These reports include a description of involvement by Dr. Michael Deem, a professor of bioengineering at Rice University. This research raises troubling scientific, legal and ethical questions.  Rice offers the following statement:

  1. Rice had no knowledge of this work.

  2. To Rice’s knowledge, none of the clinical work was performed in the United States.

  3. Regardless of where it was conducted, this work as described in press reports, violates scientific conduct guidelines and is inconsistent with ethical norms of the scientific community and Rice University.

  4. We have begun a full investigation of Dr. Deem’s involvement in this research.

And two founders of CRISPR were also quick to note their own problems with the China embryo project.

The Broad Institute’s Feng Zhang, a co-inventor of the technology, had this to say:

Although I appreciate the global threat posed by HIV, at this stage, the risks of editing embryos to knock out CCR5 seem to outweigh the potential benefits, not to mention that knocking out of CCR5 will likely render a person much more susceptible for West Nile Virus. Just as important, there are already common and highly-effective methods to prevent transmission of HIV from a parent to an unborn child.

Given the current early state of genome editing technology, I’m in favor of a moratorium on implantation of edited embryos, which seems to be the intention of the CCR5 trial, until we have come up with a thoughtful set of safety requirements first.

Not only do I see this as risky, but I am also deeply concerned about the lack of transparency surrounding this trial.

Image: Jiankui He. THE HE LAB via YOUTUBE

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Recursion Pharmaceuticals Salt Lake City, UT
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